Trial Outcomes & Findings for A Phase IV Post Approval Clinical Study of ExAblate Treatment of Metastatic Bone Tumors for the Palliation of Pain (NCT NCT01833806)

Results Overview

The statistical hypothesis is that the proportion of responders will be significantly greater than the proportion of subjects experiencing pain progression (worsened pain or increased pain medications usage). Using the Numerical Rating Score (NRS) for pain (0 being no pain and 10 being worst imaginable pain), subjects were rated as a Responder or as having Pain Progression. A responder is defined as a subject with a reduction in NRS worst score from baseline of two (2) or more points, and no increase in pain medication use. Pain Progression was increased pain of two (2) or more points.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

32 participants

Primary outcome timeframe

Three months post treatment

Results posted on

2023-04-07

Participant Flow

Participant milestones

Participant milestones
Measure	ExAblate Test Arm Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Overall Study STARTED	32
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	ExAblate Test Arm Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Overall Study Alternative treatment	2
Overall Study Death	5
Overall Study Physician Decision	1
Overall Study Lost to Follow-up	1

Baseline Characteristics

A Phase IV Post Approval Clinical Study of ExAblate Treatment of Metastatic Bone Tumors for the Palliation of Pain

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ExAblate Test Arm n=32 Participants Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Age, Continuous	58.4 years STANDARD_DEVIATION 11.4 • n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	17 Participants n=5 Participants
Race/Ethnicity, Customized White	26 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic	2 Participants n=5 Participants
Race/Ethnicity, Customized Black	1 Participants n=5 Participants
Race/Ethnicity, Customized Asian	3 Participants n=5 Participants
Region of Enrollment United States	32 participants n=5 Participants
Numerical Rating Scale	6.0 units on a scale STANDARD_DEVIATION 1.71 • n=5 Participants

PRIMARY outcome

Timeframe: Three months post treatment

Population: The analysis was based on 31 treated subjects that completed at least one follow up visit; 23 out of 31 subjects were either Responders or had Pain Progression. Eight subjects reported no change in pain.

The statistical hypothesis is that the proportion of responders will be significantly greater than the proportion of subjects experiencing pain progression (worsened pain or increased pain medications usage). Using the Numerical Rating Score (NRS) for pain (0 being no pain and 10 being worst imaginable pain), subjects were rated as a Responder or as having Pain Progression. A responder is defined as a subject with a reduction in NRS worst score from baseline of two (2) or more points, and no increase in pain medication use. Pain Progression was increased pain of two (2) or more points.

Outcome measures

Outcome measures
Measure	ExAblate Test Arm n=31 Participants Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Proportion of Responders Responders	18 Participants
Proportion of Responders Pain Progression	5 Participants
Proportion of Responders No Change	8 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Three months post treatment

Population: There were 31 subjects treated that provided a baseline score and a final follow-up score.

Patient pain intensity on an 11-point Numerical Rating Scale from 0 to 10 in which 0 is no pain and 10 is the worst pain you can imagine. The NRS was administered pre-treatment and post-treatment. The trajectory of change was presented graphically.

Outcome measures

Outcome measures
Measure	ExAblate Test Arm n=31 Participants Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Numerical Rating Scale (NRS) for Pain Score 1 Month Visit	3.8 score on a scale Interval 2.93 to 4.65
Numerical Rating Scale (NRS) for Pain Score 2 Month Visit	3.5 score on a scale Interval 2.56 to 4.54
Numerical Rating Scale (NRS) for Pain Score Baseline	5.9 score on a scale Interval 5.28 to 6.43
Numerical Rating Scale (NRS) for Pain Score 3 Month Visit	3.5 score on a scale Interval 2.31 to 4.66

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, 1 Month post treatment, 2 Month post treatment, 3 Month post treatment.

Population: One out of the 31 treated subjects did not complete the Baseline QoL (pain interference assessment and could not be included in the QoL analysis).

The BPI-QOL questionnaire is designed to show the severity and interference of pain in the lives of patients. This is a 7-item questionnaire that asks respondents the extent to which pain interferes with their general activity, mood, walking ability, normal work, relations with other people, sleep and enjoyment of life using a 0-10 numerical rating scale in which 0 represents 'does not interfere' and 10 indicates 'completely interferes'. Responses to the 7 items are averaged to form the pain interference scale score. Thus, the minimum possible is 0 and the maximum possible is 10 points. Lower scores are better, showing less interference in daily activities while higher scores show more interference and hence worse outcomes.

Outcome measures

Outcome measures
Measure	ExAblate Test Arm n=30 Participants Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Quality of Life (QOL) as Measured by Change in Brief Pain Inventory - Interference (BPI) From Baseline Baseline	5.0 score on a scale Interval 4.24 to 5.78
Quality of Life (QOL) as Measured by Change in Brief Pain Inventory - Interference (BPI) From Baseline 1 Month Visit	3.4 score on a scale Interval 2.43 to 4.3
Quality of Life (QOL) as Measured by Change in Brief Pain Inventory - Interference (BPI) From Baseline 2 Month Visit	3.2 score on a scale Interval 2.13 to 4.17
Quality of Life (QOL) as Measured by Change in Brief Pain Inventory - Interference (BPI) From Baseline 3 Month Visit	3.1 score on a scale Interval 2.08 to 4.05

Adverse Events

ExAblate Test Arm

Serious events: 1 serious events

Other events: 6 other events

Deaths: 5 deaths

Serious adverse events

Serious adverse events
Measure	ExAblate Test Arm n=32 participants at risk Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Vascular disorders Hypotension	3.1% 1/32 • Number of events 1 • Adverse events were collected for 3 months following intervention. All device or procedure related adverse events were collected

Other adverse events

Other adverse events
Measure	ExAblate Test Arm n=32 participants at risk Focused Ultrasound Surgery delivered by ExAblate MRgFUS ExAblate MRgFUS: Focused Ultrasound Ablation
Surgical and medical procedures Sonication Pain	6.2% 2/32 • Number of events 2 • Adverse events were collected for 3 months following intervention. All device or procedure related adverse events were collected
Skin and subcutaneous tissue disorders Skin burn	3.1% 1/32 • Number of events 1 • Adverse events were collected for 3 months following intervention. All device or procedure related adverse events were collected
Skin and subcutaneous tissue disorders Skin peeled	3.1% 1/32 • Number of events 1 • Adverse events were collected for 3 months following intervention. All device or procedure related adverse events were collected
Skin and subcutaneous tissue disorders Blistering	3.1% 1/32 • Number of events 1 • Adverse events were collected for 3 months following intervention. All device or procedure related adverse events were collected
Skin and subcutaneous tissue disorders Sore lip	3.1% 1/32 • Number of events 1 • Adverse events were collected for 3 months following intervention. All device or procedure related adverse events were collected

Additional Information

Nadir Alikacem, VP Global Regulated Clinical Affairs & CRO

Insightec

Phone: (214) 630-2000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place